Background: A fifth of all pediatric liver transplants in the US are performed for metabolic disease. Propionic acidemia, an autosomal recessive branched chain organic acidemia affects 1: 35000 newborns in the US, incurs a lifelong risk of encephalopathy and stroke, cardiomyopathy and infections, and is an emerging indication for LTx. Purpose/Methods: To review post-LTx survival outcomes for this rare disease, and causes of graft failure and death. Data sources were the SRTR for the US, 1988-2015 (n=38), and published non-US case series by Vara et al, 2011; Kasahara et al, 2011; Charbit et al, 2015; and Ryu et al, 2013, n=26). Results: In 64 combined primary LTx recipients from SRTR and selected non-US series, median recipient age was 27 months (range 3-156). Organ type segmental: whole was 30: 34 (69% whole liver in the US and 73% segmental in non-US series, p=0.011). Donor type living: cadaveric was 8: 56 (100% cadaveric in the US, p<0.001). Mean+/-SEM graft and patient survival was 4691+/-545 days and 5812+/-460 days, respectively. Graft loss occurred in 17 children (26%) due to primary graft failure-8 and vascular thromboses-7. Twelve of these children died, three after retransplantation. Four additional deaths occurred due to stroke-1, multiple organ failure/sepsis-2, and unknown causes-1. At 1, 5 and 10 years, actuarial graft and patient survival was 72%, 59% 59%, and 80.5%, 75%, 75%, respectively (Kaplan-Meier). LTx were distributed in three time-based eras: six in 1987-96, eight in 1997-2005, and 50 in 2006-2014. In univariate proportional hazards and multivariate analyses, the most recent of three eras demonstrated fewer graft losses (p=0.028, HR=0.492 and p=0.006, HR=0.314, respectively) and deaths (p=0.004, HR=0.564 and p=0.004, HR=0.564, respectively) compared with previous eras. Actual survival improved significantly in the most recent of three eras for graft (80% vs 37% vs 50%, p=0.017) and patient (88% vs 37% vs 67%, p=0.004) compared with previous eras. Conclusions: Liver transplantation is increasingly used to treat propionic acidemia with acceptable survival. The relatively recent use of LTx for this rare disease, and limitations of public transplant registries argue for multi-center observational follow-up of this rare transplant population to better understand the effect of LTx on neurocognitive function, and other disease-specific morbidity.

CITATION INFORMATION: Celik N, Soltys K, Bond G, Sun Q, Vockley G, Sindhi R, Mazariegos G. Liver Transplantation for Propionic Acidemia: A Review of the United States Scientific Registry for Transplant Recipients (SRTR) and Non-US Case Series. Am J Transplant. 2016;16 (suppl 3).

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